By broker is Fidelity - other than a couple sentences about the withdrawal, they provide no other information on why or other factors. I think this is standard procedure for a company that is late with audited statements, but I'm not sure?
The PR said they withdrew the preliminary B- rating on their proposed $225M notes issue, which was not completed and they also withdrew their B "long term corporate credit" rating, so I suppose that refers to existing long-term debt?
What a clueless statement - injection site irritation not a "meaningful" side effect. Irritation or inflammation of the injection site is a sign the body is reacting negatively to the substance being injected - inflammatory responses can lead to serious auto-immune complications in the long run. For a drug that must be taken for the rest of the patient's life, how could that not be "meaningful"?
It is surprising the premium is that high for a December call - there is a greater risk of having no announcement by then vs having a negative trial result reported, IMO. Perhaps they are counting on the gradual run-up and that the shares will be closer to $10 by December, whether there is an announcement or not?
How does $500M of revenue translate into $11 per share of earnings - it cost nothing to make the drug, not to mention all the ongoing R&D and administrative costs?
bergy - I admit I don't read every word you have written, but you spend an awful amount of time explaining or rationalizing posts that you have made earlier because someone misinterpreted your post or didn't catch your meaning. So, when you explain that I missed the point because your post wasn't even in the context of Etep or Drisa, when the whole point of this MB and the bulk of the recent threads are all about Etep vs Drisa, really? Perhaps you should stay more focused and be more careful about what you post instead of blaming everyone else who misses your point. "Seriously", what is the point of posting something on this board if it is not in the context of Etep's clinical value?
Why does neo have so few brain cells but posts hundreds of inane messages? What's that all about?
beaver - this is the 2nd time you've used sales in a PE computation - $720M of sales would not translate into $14 of earnings per share - you're ignoring all the expenses of manufacturing and distributing the drug, let alone all the R&D, executive and admin costs.
Are you dismissing your estimated $29K annual cost number as being too egregious and not realistic? Did you even read the press release - current treatment regimes are "palliative" (i.e. treating symptoms but not changing the course of the disease) and cost an estimated $10,000-$15,000 per month. If this drug changes the course of the disease and reduces the need for palliative treatment, it will be a cost saver. My guess is that they will price this in the $3,000-$5,000 per month, which is a bargain compared to a lot of drugs for rare diseases - many cost $20,000 per month+.
thig - the acceptance/filing of an NDA has nothing to do with the merits of the clinical evidence, only that the requisite components of the required filing are complete. I think the spineless/clueless FDA officials are counting on the AdCom to do their dirty work for them when it comes to Drisa.
So, you're claiming the ACTIVE ingredient in Scioderm's drug is a common component of cosmetics - do you really think JC is stupid enough to commit $1B for a drug that doesn't have a unique/patented mechanism of action?
Who has ever argued there will be a correlation between walking distance and dystrophin production - the only ones to argue that are the clueless shorts that don't understand the disease.
ru - What is the correlation factor for the two boys who lost ambulation - what about for the boys who are already in wheel chairs? This has been hashed out at great length on this MB, and you know it. The most important factor in determining 6MWT decline is how far the disease has progressed (i.e. what % of functional leg muscle they have left) before the boy starts treatment, which is something that is impossible to measure precisely. Another critical factor is how much the boy grows after starting treatment - will his remaining functional muscle mass be able to grow and keep pace with his body weight. Theoretically, if you start a boy early enough and salvage a major portion of his leg muscles, you may have some correlation down the road, but the idea that you will derive a correlation in boys on the cusp of losing ambulation is just senseless.
The impressive thing about the pictures is how horrible this disease must be, so any treatment that provides relief will be fast-tracked.
grey - as much as I'd like to believe it, I think orphan designation and patent issues are two separate things. Granted, given the completely different delivery mechanisms of Drisa and Etep, I can't imagine why there would be a patent issue, but that won't prevent BMRN from suing SRPT, regardless of what authorization the EMA may grant Etep?
I think it is because even though the data is good, the FDA seems to have an issue with the company's sublingual delivery mechanisms. The data for Zalviso was exceptional, but the FDA is flaking out on the delivery technology, even though it would be much safer than IV morphine pumps, which is what most hospitals currently use. I think they are worried that it would be too easy to steal and abuse?
This trial was in younger patients (2 years younger than SRPT's boys) and not comparable. Younger DMD boys are known through the natural history data to continue increasing on their 6MWT as they grow - nobody is going to believe that 25wk data on a group of boys this young is meaningful.
I don't think there is any chance that the FDA will be able to postpone the approval of Etep beyond early next year - the political pressure from the parent groups will be too great. The only way Etep gets postponed is if they get a negative AdCom opinion and the only way that happens is if they are charged with determining whether it should have full approval - Etep clearly meets the criteria for accelerated/conditional approval. Who knows what the AdCom will determine for Drisa - with no dystrophin data, cherry-picked 6MWT data and a horrible safety profile (for a chronic treatment), it is hard to imagine a positive opinion?
At this stage of the game you typically see a poster or two trying to piece together the OS number based on the few facts at hand. I found it interesting that the CEO made a point of noting that by year-end, they will be two years from finishing the enrollment in the STS trial. Without knowing how the enrollment was paced and solid assumption on the expected control arm survival, it is impossible to make a solid guess on the median survival of the treated arm, but that usually doesn't keep the speculation at bay.
For those interested in the details, the STS trial began enrollment on 9/30/11 and completed enrollment of the 620 patients (randomized 1:1) on 12/30/13. The historical survival for the control arm (Dox only) they have always referenced in presenting the Phase II results was 12.8 months and the Phase III trial is supposedly powered to detect a 33% improvement over the control arm survival. The twist in the story is that the original design of the trial only had 450 patients, but then in July 2013 they announced a design change boosting the enrollment to 620 in order to account for a higher-than-originally-expected potential survival in the control arm (based on more recent studies of survival in patients treated with Dox alone). They never have disclosed what they expect that control arm number to be, so that is where the guesswork comes in. It is worth noting that when they changed the trial design in July 2013 they projected that the 434 "events" would be reached by the first half of 2015, so they have missed that target by roughly 6 months.
Bottom line - it would appear that a significant portion of the STS trial patients are living longer than anticipated - which arm are you betting on?